Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus

Wagner, John A.; Reynolds, Thomas; Moran, Mary Lynn; Moss, Richard B.; Wine, Jeffrey J.; Flotte, Terence R.; Gardner, Phyllis

doi:10.1016/s0140-6736(05)77740-0

Cited by 209 publications

(117 citation statements)

References 3 publications

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“…It is important to note that the clinical experience with hAAT protein replacement indicates that it is safe and non-immunogenic. [21][22][23] While this mode of gene delivery may be very useful as therapy for their lung disease, the minority of patients who also suffer from liver disease will require a higher percentage of hepatocytes to be transduced. A recent study showed that the vast majority of hepatocytes took up rAAV vector, but only a small and changing subpopulation of hepatocytes were permissive for stable transduction.…”

Section: Discussionmentioning

confidence: 99%

Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors

et al. 2001

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Section: Discussionmentioning

confidence: 99%

Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors

et al. 2001

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“…13 Currently, the first phase I clinical trials using recombinant AAV are under way for cystic fibrosis. 14,15 rAAV is most often generated by cotransfection of rAAV vector plasmid and wild-type (wt) AAV helper plasmid into Ad-infected 293 cells. 16 Recent improvements in AAV helper design 17 as well as construction of non-infectious mini-Ad plasmid helper [18][19][20] have eliminated the need for Ad infection and improved the yield of rAAV per transfected cell in a crude lysate.…”

Section: Introductionmentioning

confidence: 99%

Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield

et al. 1999

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Conventional methods for rAAV purification that are based vector that is more than 99% pure. More importantly, the on cesium chloride ultracentrifugation have often produced new purification procedures consistently produce rAAV vector preparations of variable quality and resulted in sigstocks with particle-to-infectivity ratios of less than 100, nificant loss of particle infectivity. We report here several which is significantly better than conventional methods. novel purification strategies that involve the use of non-The new protocol increases the overall yield of infectious ionic iodixanol gradients followed by ion exchange or heprAAV by at least 10-fold and allows for the complete purifiarin affinity chromatography by either conventional or cation of rAAV in 1 working day. Several of these methods HPLC columns. These methods result in more than 50%should also be useful for large-scale production. recovery of rAAV from a crude lysate and routinely produce

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“…It is known that CFTR function can be restored in CF cell lines by delivering full length wild-type CFTR cDNA. [16][17][18][19][20] Studies in CF mouse models 21,22 and in CF patients [23][24][25] suggest that similar correction may also be possible in vivo. However, the conventional approach of delivering full length wild-type CFTR cDNA has encountered problems as the cDNA is at or slightly larger than the packaging size of the leading vector for CF gene therapy.…”

Section: Introductionmentioning

confidence: 99%

Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing

Mansfield¹,

Kole

Puttaraju³

et al. 2000

Gene Ther

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Most messenger RNA precursors (pre-mRNA) undergo cissplicing in which introns are excised and the adjoining exons from a single pre-mRNA are ligated together to form mature messenger RNA. This reaction is driven by a complex known as the spliceosome. Spliceosomes can also combine sequences from two independently transcribed pre-mRNAs in a process known as trans-splicing. Spliceosome-mediated RNA trans-splicing (SMaRT) is an emerging technology in which RNA pre-therapeutic molecules (PTMs) are designed to recode a specific pre-mRNA by suppressing cis-splicing while enhancing trans-splicing between the PTM and its premRNA target. This study examined the feasibility of SMaRT as a potential therapy for genetic diseases to correct mutations using cystic fibrosis (CF) as an example. We used several versions of a cystic fibrosis transmembrane conduc-

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Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus

Cited by 209 publications

References 3 publications

Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors

Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors

Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield

Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing

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